20 YEARS OF COMMITMENT 2017 ANNUAL REPORT | Translating SCIENCE • Transforming LIVES OUR COMMITMENT Make every day count At PTC, patients are at the center of everything we do. We have the opportunity to support patients and families living with rare disorders through their journey. We know that every day matters and we are committed to making a difference. OUR SCIENCE Our scientists are finding new ways to regulate biology to control disease We have several scientific research platforms focused on modulating protein expression within the cell that we believe have the potential to address many rare genetic disorders. OUR PEOPLE Care for each other, our community, and for the needs of our patients At PTC, we are looking at drug discovery and development in a whole new light, bringing new technologies and approaches to developing medicines for patients living with rare disorders and cancer. We strive every day to be better than we were the day before. At PTC Therapeutics, it is our mission to provide access to best-in-class treatments for patients who have an unmet need. We are a science-led, global biopharmaceutical company focused on the discovery, development and commercialization of clinically-differentiated medicines that provide benefits to patients with rare disorders. Founded 20 years ago, PTC Therapeutics has successfully launched two rare disorder products and has a global commercial footprint. This success is the foundation that drives investment in a robust pipeline of transformative medicines and our mission to provide access to best-in-class treatments for patients who have an unmet medical need. As we celebrate our 20th year of bringing innovative therapies to patients affected by rare disorders, we reflect on our unwavering commitment to our patients, our science and our employees. We have continually reinvested in our innovative science platforms adding to our robust pipeline in rare disorders as well commercializing two products for the treatment for Duchenne muscular dystrophy. Building on this foundation, we look forward to the next 20 years remaining loyal to our culture and our commitment to make every day count for patients. 2 3 A Patient’s Story “ If I could say one thing to PTC it would be this: Thank you for not giving up and throwing in the towel. It truly is working. Everything he (Benjamin) can do now is BENJAMIN RODRIGUEZ because of ataluren.” -Diana Rodriguez Birthday parties shouldn’t be a reason to worry for any parent but when Diana Rodriguez and her son were invited It was an achievement she never thought possible. Since to one at a new community park, the mother of two July 2016, Benjamin has been enrolled in a clinical trial paused. Benjamin, 5, has Duchenne muscular dystrophy and takes ataluren. His parents soon noticed a difference and outdoor parties are prime opportunities for kids to run when Benjamin, who use to just walk fast, began to run. around and, well, be kids. Then, one day, he jumped. He now attends school and Diana often feared her son would be inadvertently knocked participates in physical education classes, running, and down and hurt or feel left out if he couldn’t participate in playing soccer, just like any other 5-year-old. an activity. Adding to her angst was the party venue: A new “I definitely didn’t think he’d be running around.” Diana community park full of playground equipment that could said. “He still has all this energy and now he uses it to prove very challenging for a young boy with DMD. climb on things and run around. And, he keeps asking to But on this sunny Florida day in February, it didn’t take go back to the playground.” long for Diana to realize her worries were unfounded. Ben It’s a remarkable improvement for a little boy who, walked up to a new structure and started to climb on it. according to his mom, remembers there was a time he Later, she saw him on a zipline. couldn’t do things like other boys. That’s not the case “He was feeling good about himself,” she said. “It had to anymore for Benjamin, who loves Star Wars and has his do with his confidence. I could see it, and that meant I own light saber. could step back a bit because he was showing me that he “We are so lucky to have ataluren,” Diana said. “If I could do it. He was OK.” could say one thing to PTC it would be this: Thank you Hours later, Diana logged onto her Twitter account and for not giving up and throwing in the towel. It truly is announced: Today was the first time we went to the park working. Everything he (Benjamin) can do now is because and Benjamin didn’t hesitate to play. of ataluren.” 1998 1999 • PTC incorporated • Tularik collaboration Milestones • Series A&B financing • Initiate nonsense • PTC124 (ataluren/Translarna™) discovery suppression program 2 3 A Scientist’s Story ANU BHATTACHARYYA, DIRECTOR OF BIOLOGY PTC Just because a collaboration ends doesn’t mean a scientist stops thinking about it, and Anu Bhattacharyya couldn’t stop thinking about the brain and the diseases that affect it. She wanted to know more about the mechanisms that lead to neurological disorders so she invested in an atlas “ This is a very exciting time for emerging of the brain and studied it to learn more about the organ’s research in the Huntington’s disease different regions and why they break down in some people. community, and PTC is committed to The more she learned, the stronger her passion grew. following the science to discover and She started to wonder whether she could harness her develop novel small molecule treatments passion into something that might help people afflicted with Huntington’s disease, a fatal genetic disorder that for patients with rare diseases.” causes the progressive breakdown of nerve cells in the -Anu Bhattacharyya brain. It deteriorates a person’s physical and mental abilities during their prime adult years and has no cure. With that thought in mind and a business case in hand, she walked into her boss’ office to pitch her idea, confident that “This is a very exciting time for emerging research in the it was the right thing to do. He agreed. Huntington’s disease community, and PTC is committed to following the science to discover and develop novel Go for it, he told her. small molecule treatments for patients with rare diseases,” She got support from PTC’s leadership team even Bhattacharyya said. though resources were scarce and the company didn’t She built a business case that she believed would prove have a Huntington’s program. Combine that endorsement that a Huntington’s program could fit into PTC’s business with the unmet medical need and the result is a recipe goals and strategy of innovation, and, eventually, its that led to PTC’s Huntington’s disease program, which splicing platform. recently announced a research collaboration with the CHDI Foundation. “Huntington’s disease is a rare genetic disorder so it falls right into PTC’s sweet spot,” she said. “One appealing “When I started to build the rationale for the discovery feature of Huntington’s disease is that it’s a monogenic and development of this program, I didn’t know the disorder and the key culprit is a single expansion mutation Huntington’s research and patient community,” in the huntingtin gene. That makes it an easier problem to Bhattacharyya said. “Now, our relationships are strong tackle; we know the key cause of the disease, so targeting and our discovery program is showing great promise to the expression of huntingtin is the most direct and efficient deliver an oral medication for Huntington’s disease. way of targeting Huntington’s disease.” That is what can be accomplished at PTC.” 2000 2001 2002 • Moved into New Jersey • Series D financing • PTC124 (ataluren/Translarna™): corporate headquarters Lead optimization • Series C financing 4 5 Strong commercial growth Translarna™ & Emflaza™ Duchenne Net Sales ($ in millions) “ Every day, PTC scientists 180 dedicate their efforts to TM Emflaza finding new and better (deflazacort) treatments for people with 144 TM rare disorders and oncology. Translarna (ataluren) We take special pride in the innovation seen throughout 108 our pipeline and that’s because we developed it in-house; it’s a reflection 72 of our hard work and dedication to positively impact the lives of patients 36 and their families.” Dr. Nikolai Naryshkin VP, Biology 0 2014 2015 2016 2017 2003 2004 2005 • Facility expansion • Series E financing • Facility expansion • NIH STTR grant: Nonsense suppression • Series E-2 financing • PTC124 (ataluren/Translarna™) IND filed • PTC124 (ataluren/Translarna™) • PTC124 (ataluren/Translarna™) Phase 1 Phase 2a (004) Duchenne healthy volunteer study 4 5 Our Pipeline PRODUCT/ DISCOVERY PRECLINICAL PHASE 1 PHASE 2 PHASE 3 COMMERCIAL PLATFORM ORPHAN GENETIC DISORDERS EMFLAZATM DMD (deflazacort) TM Translarna DMD (ataluren) nonsense readthrough * Aniridia Dravet / CDKL5 Next Generation nonsense readthrough SMA SMA alternative splicing Huntington’s HD alternative splicing Familial Dysautonomia FD alternative splicing ONCOLOGY PTC596 BMI1 tumor stem cell targeting DHODH (1) Orphan Oncology DHODH (2) *Marketing authorization has specific obligation to conduct additional nmDMD trial and requires annual renewal following reassessment by EMA 2006 2007 2008 • SMA Foundation Grant • CELG Equity investment & collaboration • (ataluren/Translarna™) • FDA Grant: PTC124 (ataluren/Translarna™) • Nature publication – PTC124 Phase 2b (007) Duchenne Duchenne (ataluren/Translarna™) nonsense platform • Fight SMA grant • JCP publication – HV study – PTC124 (ataluren/Translarna™) • SMA Foundation grant 6 7 A Message to Our Shareholders This acquisition was a strategic opportunity for PTC; with our long-standing partnership with the Duchenne commu- nity, we were uniquely positioned to bring this medicine to patients in the U.S.